The EU Clinical Trials Register currently displays
33591
clinical trials with a EudraCT protocol, of which
5439
are clinical trials conducted with subjects less than 18 years old.
The register also displays information on
18700
older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

Search Tips:
Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.

The primary objective of the study is to assess the efficacy of rhNGF eye drops solution administered over 1 month versus a vehicle control in patients with macular edema associated with retinitis pigmentosa

E.2.2

Secondary objectives of the trial

The secondary objective of this study is to assess safety and tolerability of hNGF eye drops solution administered over 1 month versus a vehicle control in patients with macular edema associated with retinitis pigmentosa

E.2.3

Trial contains a sub-study

No

E.3

Principal inclusion criteria

1. Patients between 18 and 60 years of age.
2. Patients with typical orms of RP characterized by the following
clinical features:
a) classic fundus appearance (i.e. intraretinal pigment deposits,thinning and atrophy of the RPE in the mid- and far peripheral retina, with relative RPE preservation in the macula, waxy
pallor of the optic disc, attenuation of the retinal vessels)
b) reduced and delayed ERG responses
c) visual field constriction
and/or atypical forms of RP:
The atypical form of RP is characterized by early onset (first decade) or syndromic forms of RP (eg paravenous sector pericentral or unilateral RP, Leber congenital amaurosis, Resfum disease, Usher syndrome, Bardet - Biedl, etc. ).
3. presence of macular edema documented by OCT (macular thickness greater than 250 um) lasting for at least 3 months and not in treatment for at least 1 month;
4 Best corrected distance visual acuity (BCDVA) between 20/100 (2/10 or 0.1 logMAR) and 20/25 (8/10 or +0.7 logMAR) , near vision lower than second character in either eye.
5. Absence of other confounding ocular diseases.
6. Only patients who satisfy all Informed Consent requirements may be
included in the study. The patient and/or his/her impartial witness must
read, sign and date the Informed Consent document before any
studyrelated procedures are performed. The Informed Consent form signed by patients and/or impartial witness must have been approved by the IEC for the current study.
7. Patients must have the ability and willingness to comply with study
procedures.

E.4

Principal exclusion criteria

1. Presence of macular edema associated with diabetes , choroidal neovascularization or after eye surgery.
2 . Patients with diabetes mellitus
3 . History of any ocular surgery (including laser or refractive surgical
procedures) in either eye within the 90 days before study enrolment.
Ocular surgery will not be allowed during the study treatment period and
elective ocular surgery procedure.
4. Evidence of an active ocular infection.
5. History of uveitis or evidence of intraocular inflammation in either eye.
6. History or evidence of glaucoma or an IOP greater than or equal 21
mmHg in either eye at the time of study enrollment
7. Anterior segment abnormalities or media opacities obscuring the view
of the posterior pole in either eye.
8. Treatment with corticosteroids (systemic, periocular or intravitreal)
) in either eye within 90 days of study enrollment.
9. Use of any topical medication other than the study medication for the
treatment of ocular diseases with the exception of artificial tears during the study period.
10. Presence or history of any ocular or systemic disorder or condition
that might significantly limit visual acuity or the visual field, hinder the
efficacy of the study treatment or its evaluation, could possibly interfere
with the interpretation of study results, or could be judged by the investigator to be incompatible with the study visit schedule or conduct of trail procedures.
11. Known hypersensitivity to one of the components of the study or
procedural medications.
12. Participation in another clinical study at the same time as the present study or within 90 days of screening/baseline visit.
13. History of drug, medication or alcohol abuse or addiction.
14. Females of childbearing potential (those who are not surgically
sterilized or post-menopausal for at least 1 year) are excluded from participation in the study if they meet any one of the following conditions:
a. are currently pregnant or,
b. have a positive result on the urine pregnancy test at the Screening/Baseline Visit or,
c. intend to become pregnant during the study treatment period or,
d. are breast-feeding or,
e. not willing to use highly effective birth control measures, such as:
Hormonal contraceptives – oral, implanted, transdermal, or injected
and/or mechanical barrier methods – spermicide in conjunction with a
barrier such as a condom or diaphragm or IUD during the entire course
of and 30 days after the study treatment periods.

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint will be to evaluate the effects of NGF eye drops on macular thickness in patients with RP associated with macular edema. Macular thickness greater than 250μm will be considered significant. Treatment will be considered effective if it will induce an improvement of at least one of the following parameters: 1) macular thickness less than 250μm; 2) visual acuity of at least 20 EDTRS letters; 3) near visual acuity of at least 1 line

E.5.1.1

Timepoint(s) of evaluation of this end point

after one month of experimental treatment

E.5.2

Secondary end point(s)

The secondary endpoints of this study are to evaluate differences between the two treatment groups of the following parameters:
- Best-corrected visual acuity for distance
- best-corrected visual acuity for near
-contrast sensitivity
- visual-field
-OCT
- ERG
-Quality of Life (NEI-VFQ)
- Number of drop out for inefficacy
-number of recurrence of macular edema

E.5.2.1

Timepoint(s) of evaluation of this end point

secondary endpoints will be evaluated at the end of experimental treatment (1 month) and at 3, 6 and 12 months of follow-up

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

No

E.6.2

Prophylaxis

No

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

No

E.6.7

Pharmacodynamic

No

E.6.8

Bioequivalence

No

E.6.9

Dose response

No

E.6.10

Pharmacogenetic

No

E.6.11

Pharmacogenomic

No

E.6.12

Pharmacoeconomic

No

E.6.13

Others

No

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

No

E.7.1.1

First administration to humans

No

E.7.1.2

Bioequivalence study

No

E.7.1.3

Other

No

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

No

E.7.4

Therapeutic use (Phase IV)

No

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

No

E.8.1.3

Single blind

No

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

No

E.8.1.7

Other

No

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

No

E.8.2.2

Placebo

Yes

E.8.2.3

Other

No

E.8.2.4

Number of treatment arms in the trial

2

E.8.3

The trial involves single site in the Member State concerned

No

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

2

E.8.5

The trial involves multiple Member States

No

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

No

E.8.6.2

Trial being conducted completely outside of the EEA

No

E.8.7

Trial has a data monitoring committee

No

E.8.8

Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

1

E.8.9.1

In the Member State concerned months

6

E.8.9.1

In the Member State concerned days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

No

F.1.1.1

In Utero

No

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

No

F.1.1.3

Newborns (0-27 days)

No

F.1.1.4

Infants and toddlers (28 days-23 months)

No

F.1.1.5

Children (2-11years)

No

F.1.1.6

Adolescents (12-17 years)

No

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

45

F.1.3

Elderly (>=65 years)

No

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

No

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

No

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

No

F.3.3.4

Nursing women

No

F.3.3.5

Emergency situation

No

F.3.3.6

Subjects incapable of giving consent personally

No

F.3.3.7

Others

No

F.4 Planned number of subjects to be included

F.4.1

In the member state

45

F.5

Plans for treatment or care after the subject has ended the participation in the trial
(if it is different from the expected normal treatment of that condition)

None

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned